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Working Together To Advance
Rare Diseases Research

The National Center for Advancing Translational Sciences (NCATS) Toolkit for Patient-Focused Therapy Development provides information and resources to help patient groups support the process of developing a treatment or cure for their disease(s). The goal is to ensure that patient groups are engaged as essential partners from the beginning to the end of the therapy research and development (R&D) process, whether the therapeutic approach is a drug, biologic, or medical device.

The information in NCATS Toolkit highlights tips and strategies from the collected resources and from presentations by representatives of patient groups, academia, government, and industry at conferences and public meetings, as well as  informal conversations with these same stakeholders. 

This is a living site. New resources and information will be added as they are developed for and by patient groups in concert with their academic, government, industry, and advocacy partners. You are encouraged to share any helpful resources, tips, or strategies for possible addition to NCATS Toolkit through the Contact Page.

Getting Started

The information and resources in this section can help you learn about how therapies (drugs, biologics, and medical devices) are developed, why it is important for patients to engage throughout the process, and how to build relationships with key partners.

Research and Development Process

About NCATS Toolkit

Read about how and why NCATS developed this Toolkit to help patient groups advance through the process of therapy development.